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New treatment option for Hepatocellular Carcinoma, Childhood
Official title Checkpoint Inhibition In Pediatric Hepatocellular Carcinoma
ClinicalTrials.gov ID: NCT04134559
What this study is testing
What is Pembrolizumab?
Pembrolizumab is an investigational medicine, being studied as a potential treatment for hepatocellular carcinoma, childhood.
Also referred to as Keytruda.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This research study is studying an immunotherapy drug (pembrolizumab or KEYTRUDA) as a possible treatment for pediatric hepatocellular carcinoma or hepatocellular neoplasm not otherwise specified (HCN NOS).
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 0 to 30
You may be able to join if
- Age: Patients must be \<30 years of age at the time of study enrollment.
- Diagnosis: Patients must have relapsed/refractory, histologically confirmed HCC to be eligible for enrollment. Patients with hepatocellular neoplasm...
- Disease Status: Participants must have measurable disease by RECIST criteria, defined as at least one lesion that can be accurately measured in at...
- Performance Level: Karnofsky performance status ≥ 60% for patients ≥ 16 years of age or Lansky ≥ 60% for patients \< 16 years of age.
- Prior Therapy: Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy.
You likely can't join if
- Participants who are receiving any other investigational agents are not eligible.
- Participants who have received checkpoint inhibitors (PD-1, PD-L1, and CTLA-4 inhibitors) are not eligible.
- Participants who have received antibody-based therapies are not eligible if they are within 3 half-lives of receipt of the last antibody dose.
- Participants who are receiving chronic steroids are not eligible.Chronic steroids are defined as either \> or = 2mg/kg/day of body weight or \> or =...
- Participants who are receiving anti-inflammatory or immunosuppressive medications are not eligible.
- Participants with known autoimmune disease, with the exceptions of childhood asthma or atopic dermatitis, are not eligible.
See the full eligibility criteria
Who can join
- Age: Patients must be \<30 years of age at the time of study enrollment.
- Diagnosis: Patients must have relapsed/refractory, histologically confirmed HCC to be eligible for enrollment. Patients with hepatocellular neoplasm not otherwise specified (HCN NOS) will also be eligible.
- Disease Status: Participants must have measurable disease by RECIST criteria, defined as at least one lesion that can be accurately measured in at least one dimension (longest diameter to be recorded for non-nodal...
- Performance Level: Karnofsky performance status ≥ 60% for patients ≥ 16 years of age or Lansky ≥ 60% for patients \< 16 years of age.
- Prior Therapy: Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy.
- Patients must not have received standard or targeted treatment regimens within 14 days of initiation of treatment with pembrolizumab.
- Patients must not have received prior radiotherapy within 7 days of initiation of treatment with pembrolizumab. Patients who have experienced radiation-induced adverse events must recover to a grade 1 prior to...
- Organ Function Requirements: Participants must have normal organ and marrow function as defined below:
- Adequate Bone Marrow Function defined as:
- Peripheral absolute neutrophil count (ANC) ≥ 750/μL
- Platelet count ≥ 75,000/μL (can be transfused)
- Adequate Liver Function defined as:
- Total bilirubin \< 1.5 x institutional upper limit of normal (ULN)
- AST(SGOT) ≤ 2.5 x ULN
- ALT(SGPT) ≤ 2.5 x ULN
- If liver function studies are more elevated than the thresholds above, and if if this elevation is felt secondary to tumor, patients may still be eligible for enrollment after discussion with the study PI.
- Adequate Renal and Metabolic Function defined as:
- A serum creatinine based on age/gender as follows:
- Age Maximum Serum Creatinine (mg/dL) Male Female
- 1 to \<2 years 0.6 0.6
- 2 to \<6 years 0.8 0.8
- 6 to \<10 years 1.0 1.0
- 10 to \<13 years 1.2 1.2
- 13 to \<16 years 1.5 1.4
- \>16 years 1.7 1.4
- Creatinine clearance ≥60 mL/min/1.73 m2 for participants with creatinine levels above institutional normal.
- Amylase ≤ 1.5 x ULN
- Lipase ≤ 1.5 x ULN
- If liver function studies are more elevated than the thresholds above, and if this elevation is felt secondary to tumor, patients may still be eligible for enrollment after discussion with the study PI. \-- Adequate...
- Confirmation of Insurance Pre-authorization approval for Pembrolizumab.
- Patients, their parent, and/or legally authorized representative must be able to understand and be willing to sign a written informed consent document. Assent for participants \< 18 years will follow institutional...
- The effects of pembrolizumab on the developing human fetus are unknown. For this reason, patients of child-bearing and child-fathering potential must agree to use adequate contraception (hormonal or barrier method of...
- Women of child bearing potential (WOCBP) must have a negative serum or urine pregnancy test within 24 hours of each treatment.
- A tumor sample must be available for submission to the central laboratory (Dana-Farber Cancer Institute, see Section 9). If surgery was performed at the time of recurrence, this sample, in addition to a diagnostic...
What rules you out
- Participants who are receiving any other investigational agents are not eligible.
- Participants who have received checkpoint inhibitors (PD-1, PD-L1, and CTLA-4 inhibitors) are not eligible.
- Participants who have received antibody-based therapies are not eligible if they are within 3 half-lives of receipt of the last antibody dose.
- Participants who are receiving chronic steroids are not eligible.Chronic steroids are defined as either \> or = 2mg/kg/day of body weight or \> or = 20mg/day of prednisone or equivalent for persons who weigh \> or =...
- Participants who are receiving anti-inflammatory or immunosuppressive medications are not eligible.
- Participants with known autoimmune disease, with the exceptions of childhood asthma or atopic dermatitis, are not eligible.
- Patients with a history of a positive test for human immunodeficiency virus or acquired immunodeficiency syndrome are not eligible.
- Patients with a history of allergic reactions attributed to compounds of similar chemical or biologic composition to pembrolizumab are not eligible. History of severe allergy to monoclonal antibody therapies (i.e...
- Patients with uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, or psychiatric illness/social situations that would limit compliance with...
- Patients with prior solid organ transplantation are not eligible.
The study team makes the final eligibility decision.
Where it's taking place
- San Francisco, California, United States
- Boston, Massachusetts, United States
- Cincinnati, Ohio, United States
- Houston, Texas, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0 years to 30 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include San Francisco, California, United States; Boston, Massachusetts, United States; Cincinnati, Ohio, United States; Houston, Texas, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.